For Chronic Myeloid Leukemia Patient Group Advocates

leukemia

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

Philadelphia chromosome

A certain change in chromosomes (on chromosome 22) found in 95% of patients who have CML. The Philadelphia chromosome results from a mutation that involves the fusion of parts of chromosome 9 and chromosome 22 (the bcr-abl fusion gene)

haematological

Relating to blood or the formation of blood

Also called: hematological

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Chromosome

A chromosome is a structure of DNA that carries the genetic makeup in the nucleus of the cell. Chromosomes contain giant chain molecules of DNA, coiled and folded as aggregates with specific proteins. Chromosomes ensure that during cell division the hereditary information is evenly distributed to the daughter cells. Normal human body cells have 46 chromosomes. Cancer cells can have a different number and/or structure of chromosomes.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Nilotinib

Trade name: Tasigna, development name: AMN107, inhibits BCR-ABL tyrosine kinase. Authorized for marketing in the EU since 2007 for the treatment of CML and Ph+ALL.Other names: |AMN107|Tasigna

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

Ph+

Abbreviation for "Philadelphia-Chromosome-positive", meaning the presence of a certain change in chromosomes (on chromosome 22) found in 95% of patients who have CML. The Philadelphia chromosome results from a mutation that involves the fusion of parts of chromosome 9 and chromosome 22 (the bcr-abl fusion gene).

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

The Leukemia Patient Advocates Foundation is a patient-led non-profit foundation in Switzerland. Its mission is to improve the lives & survival of patients affected by Leukemia. It is a platform for discussions & best practice sharing to Leukemia patient groups worldwide. The foundation collaborates with all stakeholders involved in research, policy, treatment and care. It also acts as a platform for patient organisations – concentrating on educating, connecting and supporting patient group leaders.

CML Advocates Network

Hosted by the Leukemia Patient Advocates Foundation, the global "CML Advocates Network" (http://cmladvocates.net) connects 115 patient organisations in 86 countries on all continents. The CML Advocates Network is a worldwide network designed and run by CML patients and carers who are registered patient group advocates and organisers.

The Leukemia Patient Advocates Foundation hosts the "CML Horizons" conference as the annual global leadership meeting that welcomes patient leaders from all world regions (http://www.cmladvocates.net/cmlhorizons). An elected "CML Horizons Steering Committee", as a steering committee (as defined in the foundation's statutes) within the Leukemia Patient Advocates Foundation, carries all responsibility to run the "CML Horizons" conferences. Following elections by the worldwide CML community in May 2017, the CML Horizons Steering Committee consists of the following nine members of which six are CML patients themselves, and which represent all major regions of the world:

MPN Advocates Network

Hosted by the Leukemia Patient Advocates Foundation, the global "MPN Advocates Network" (http://www.mpn-advocates.net) was founded by representatives from the Netherlands, Spain and the UK. The MPN Advocates Network's immediate goal is to increase the international scope of the organisation by contacting MPN patient groups in other countries and inviting them to become part of the network. The initial focus will be groups in Europe, but the intention is to be a world-wide organisation.

The MPN Advocates Network hosts the "MPN Horizons", the international conference for MPN patient advocacy leaders, works on educating patient advocates, building patient groups’ capacity, implementing advocacy initiatives with a real impact in the countries and finally enforcing cooperation in the global “MPN Advocates Network”.

A Steering Committee within the Leukemia Patient Advocates Foundation carries all responsibility to run the MPN Advocates Network. The members of the Steering Committee are:

Jonathan Mathias, Chair (UK)

Cheryl Petruk (Canada)

Felice Bombaci (Italy)

Giora Sharf (Israel)

Ilse Jans (Belgium)

Peter Loffeelhardt (Spain)

Robi Zelig (Israel)

Werner Zinkand (Germany)

William Crowley (U.S.A)

Giora Sharf (Israel), as a trustee of the Leukemia Patient Advocates Foundation, supervises the finances of the MPN Advocates Network and its compliance with Swiss Regulatory requirements regarding the non-profit status.

CLL Advocates Network

Hosted by the Leukemia Patient Advocates Foundation, the establishment of the CLLAdvocates Network (CLLAN) (http://www.clladvocates.net) and commencement of the new network’s activities aims to ensure the successes seen in CML are replicated for CLLand common goals are shared. The CLLAdvocates Network (CLLAN) was founded by representatives from Canada, Czech Republic, France, Israel, Portugal, UK and the United States. The CLLAdvocates Network Steering Committee consists of the following 8 members, of which 3 are CLLpatients and one a carer. The other members are CLLpatient advocates:

The CLLAdvocates Network hosts the "CLLHorizons", a unique opportunity for CLLpatient organization representatives to meet and network, giving them the opportunity to discuss issues facing CLLpatients and hopefully start to think about what they could do better to help CLLpatients in their communities.

Acute Leukemia Advocates Network (ALAN)

The ALAN is an independent global network of patient organisations representatives and patient organisations, dedicated to

changing outcomes of patients with acute leukemias by strengthening patient advocacy in that area by developing patient information and specific support for patients with acute leukemias and their carers in all countries;

strengthening patient organisations by sharing best practices and providing toolkits in patient advocacy;

creating awareness about acute leukemias and how to better support leukemia patients; advocating for better treatment, care & access to healthcare services;

improving education for healthcare professionals serving leukemia patients as well as collaborating with other initiatives and stakeholders with similar goals.

Similar to the CML Advocates Network, MPN Advocates Network and CLLAdvocates Network within the LePAF, the ALAN is not a network mainly targeting (english-language) patients and carers directly, but acts as a “network of organisations”. It aims to build capacity in the members of the network to deliver tailored services to acute leukemia patients and carers on the national level, while joining forces between organisations on the policy and research level across countries.

The first ALAN Steering Committee consists of the following patient advocates:

Zack Pemberton-Whiteley, Chair (UK)

Sofia Sa Cardoso,Treasurer (Portugal)

Anita Waldmann (Germany)

Sophie Wintrich (UK)

Diego Villalón (Spain)

Jan Geissler (Leukemia Patient Advocates Foundation representative)

Board of the Leukemia Patient Advocates Foundation

The foundation's statutes mandate the foundation board only with supervisory and financial management roles, while its statutory committees, as listed above, are fully self-governed, autonomous initiatives. Projects are run by the committees, not by the foundation board. Formally, the Leukemia Patient Advocates Foundation is represented by the following trustees of the Foundation:

Management Team of the Leukemia Patient Advocates Foundation

Lidija Pecova - Programme Manager ()

Celia Marín - Programme Manager ()

Registered Address & Contact Details

We are purely patient-run, non-profit and public interest, and we have no legal department. Talking to each other first is always the best solution. If you see a challenge for whatever reason, please contact us directly - we are very confident any issue can be solved.

CML

The 2017 American Society of Clinical Oncology (ASCO) Annual Meeting is taking place June 2–6 in Chicago.

At the CML Education Session, CML expert Dr. Ehab Atallah, MD, associate professor of medicine in the division of hematology and oncology at the Medical College of Wisconsin, has been speaking during an Education Session about discontinuation of tyrosine kinase inhibitors in chronic myeloid leukemia (CML) presenting about the "Promise of stopping TKIs: Is it ready for prime time"?

Even though no new data was presented (see ASH 2016 Report), it has been very interesting to listen to the US perspective.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

The 2017 American Society of Clinical Oncology (ASCO) Annual Meeting were taking place June 2–6 in Chicago.

At ASCO, Lia Gore, MD, co-director of the Hematological Malignancies Program at the University of Colorado Cancer Center and pediatric oncologist at Children’s Hospital Colorado, presented exciting data about Dasatinib in pediatric CML patients: CML in children is ultra-rare, making it very difficult to study - CML cases make up only 3% of all leukemias in children.

The data presented here has been submitted to FDA and EMA for the approval in pediatric use, and is quite impressive: The side effect profile seems to be very favourable and the inhibition of bone growth in children on TKIs seems to be less of an issue in Dasatinib than in Imatinib.

However, given that administration of medicines in children and being adherent to therapy is often a challenge for young CML patients (and their parents), the availability of Dasatinib also as a powder-based suspension as well as the fact that the drug can be taken without or with food may make life much easier.

The investigators concluded that Dasatinib may be considered as a new standard of care for pediatric CML patients.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

The 2017 American Society of Clinical Oncology (ASCO) Annual Meeting were taking place June 2–6 in Chicago and our cofounder Jan Geissler has collected all the highlights of this important meeting for chronic myeloid leukemia community.

In the recent past, not only hematologists but also patient groups have expressed their concern that "stopping CML therapy out in the field" may lead to bad practice by doctors not following expert recommendations, and have suggested stopping TKI therapy should only be done in clinical trials.

Of course, especially after NCCN has updated its CML guideline and has given guidance on stopping treatment despite the prematurity of data, we could expect that this would be largely ignored out in the field.

So here is Jan Geissler's shock poster at #ASCO17: The high arts of CML therapy and the sad reality - we need to be grateful for investigators spelling it out so clearly. He is shocked, as his gut feeling about bad practice is now supported by data.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

Tyrosine kinase inhibitors (TKI) have dramatically improved survival of CML and made it a chronic disease. However, life-long therapy is still advised according to expert recommendations and the product labels of current TKI treatments. Depending on the choice of TKI, about 40-70% of CML patients reach a deep molecular response, meaning a BCR-ABL ratio of 0.01% (MR4) or below. Given it had been observed that some patients were able to stop treatment in deep remission without a recurrence of the disease, the effectiveness of stopping TKI treatment of CML patients at large having a sustained, deep molecular response is a key question that is being investigated in various studies, and is probably the most debated CML topic at this year’s ASH.

We are summarizing the most important presentations, discussions and posters on that important topic at the ASH congress this year.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

The American Society of Hematology’s annual meeting brings together hematologists from around the world to discuss critical issues in hematology, examines the latest clinical advances in this area and explores the year's most significant scientific discoveries and updates.

We are happy to share with you these short video interviews that highlight a CML patient’s view on the safety of stopping treatment in Chronic Myeloid Leukemia, the new BCR-ABL inhibitor ABL001 for CML treatment, generic Imatinib in the age of Imatinib patent expiration, patient involvement in th ASH congress, as well as some European programs and initiatives in hematology like HARMONY and the European Reference Network EuroBloodNet.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

This is the world’s most comprehensive haematology event of the year and it is very important to be ready before the meeting as CML advocates in order not to miss out on any important session about chronic myeloid leukaemia.

We are happy to share with you some key information about CML sessions hoping it will assist you to prepare and outline your days and make the most out of your time whilst at #ASH17.

Click on "Read more" and check what is offered for patient advocates, haematologists and other stakeholders interested in CML at American Society of Hematology Annual Meeting this year. If you are coming to #ASH17, don't miss any of them, they will all be really interesting!

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

THE EUPATI PATIENT EXPERT TRAINING COURSE IS TAILORED TO PATIENT ADVOCATES AND TURNS THEM INTO PATIENT EXPERTS IN MEDICINES RESEARCH AND DEVELOPMENT.

DEADLINE FOR APPLICATIONS IS 31 MARCH 2017

The EUPATI Patient Expert Training Course in Medicines Research & Development is an exciting and unique opportunity, offering patient advocates expert-level training that is specifically tailored for them.

This 14-month tailor-made course, based on e-learning with additional two face-to-face training weeks, gives you the tools as CML patient advocate leaders to be able to contribute to be part of the research and development process and all related regulatory processes. It helps to understand all processes of pre-clinical and clinical research, drug safety, benefit/risk assessment and health technology assessment at an expert level.

So far, three CML advocates members have participated in the EUPATI course: Conny Borowczak, Aimo Stromberg and Sarunas Narbutas. Read about Conny's experience by clicking on "Read more", and contact them if you have questions.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

Chronic Myeloid Leukemia is a Rare Disease

What is a Rare Disease?

The actual prevalence of rare diseases can vary between populations, making it difficult to provide a precise numerical definition. In Europe and many other regions, a disease is defined as rare when it affects fewer than 1 in 2000. One rare disease may affect only a handful of patients in the European Union and another may touch as many as 245,000. In the EU, as many as 30 million people may be affected by one of over 6000 existing rare diseases. Click here to check more information about Rare Diseases at Eurordis, Rare Diseases Europe.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

Currently, 24 clinical studies are listed that are actively recruiting patients. Another 11 studies are still ongoing but no longer including new patients.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

Equip yourself with knowledge on how to make a difference to the lives of patients, identify opportunities in patient support & advocacy, empower yourself & gain the courage to fight for the rights of CML patients, learn from the experts - both patient advocates and physicians.

This exciting one of its kind conference will enable you to be able to exchange ideas, interact & share best practice. You will also have the opportunity start networking across borders and build alliances and so much more for you to bring back to your own organization.

Registration process will start the 2nd week of January 2017 and will inform you as soon as the registration platform is online.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

Registration is now open - Join us for the 15th “CML Horizons: Learn. Share. Grow" Conference

DATE: 26TH TO 28TH MAY 2017LOCATION: FRANKFURT, GERMANY

Equip yourself with knowledge on how to make a difference to the lives of patients, identify opportunities in patient support & advocacy, empower yourself & gain the courage to fight for the rights of CML patients, learn from the experts - both patient advocates and physicians.

This exciting one of its kind conference will enable you to be able to exchange ideas, interact & share best practice. You will also have the opportunity start networking across borders and build alliances and so much more for you to bring back to your own organization.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

Registration is now open - Join us for the 16th “CML Horizons: Learn. Share. Grow" Conference

DATE: 4TH to 6th MAY 2018LOCATION: WARSAW, POLAND

Equip yourself with knowledge on how to make a difference to the lives of patients, identify opportunities in patient support & advocacy, empower yourself & gain the courage to fight for the rights of CML patients, learn from the experts - both patient advocates and physicians.

This exciting one of its kind conference will enable you to be able to exchange ideas, interact & share best practice. You will also have the opportunity start networking across borders and build alliances and so much more for you to bring back to your own organization.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

24 European Reference Networks, based on an initiative of the European Commission and EU Member States, are networks of healthcare professionals, centres of expertise and patient organisations aimed at improving quality, safety, and access to highly specialised healthcare in Europe.

The EuroBloodNet European Reference Network, covering rare haematological diseases, has kicked off on 27 January 2017 in Paris with 66 partner organisations from 15 countries. The Leukemia Patient Advocates Foundation, CML Advocates Network and CLLAdvocates Network are deeply involved in EuroBloodNet to make sure it delivers to patients' needs.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

haematological

Relating to blood or the formation of blood

Also called: hematological

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

haematological

Relating to blood or the formation of blood

Also called: hematological

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

The Innovative Medicines Initiative (IMI) has approved HARMONY, a 5-year project that aims to foster better access and care for patients with various hematologic malignancies (HM) with the use of big data. The project is made up of 51 partners from 11 European countries, including 7 pharmaceutical companies.

We are very excited that CML Advocates Network will be contributing to this unique project. Jan Geissler is the Work Package leader that will be coordinating HARMONY's Stakeholder Forum and patient input of the haematology patient community into this project. The project will kick-off on 16 January 2017 in Salamanca, Spain.

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

haematological

Relating to blood or the formation of blood

Also called: hematological

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

In this section, we are collecting and publishing news focused on treatment-free remission in CML on a regular basis. We offer you to check the "News on TFR" section to be informed about latest advances in stopping treatment for CML patients.

Abstract

BCR-ABL1 tyrosine kinase inhibitors have dramatically improved outcomes for patients with chronic myeloid leukemia, and current studies are investigating whether some patients may be able to suspend therapy yet maintain response in a state known as “treatment-free remission” (TFR). Results from ongoing studies suggest that ≈ 40% to 60% of patients in sustained (generally ≥ 2 years) deep molecular response (defined as a 4-log or deeper reduction in BCR-ABL1 transcripts, depending on the study) who attempt TFR may successfully remain off treatment. Results from TFR clinical trials, patient considerations for attempting TFR, and potential predictive factors associated with successful TFR are reviewed herein.

FDA updates the label of Tasigna to reflect that certain patients with a type of leukaemia that may be eligible to stop treatment after sustained response

Discontinuation in treatment marks a first in Chronic Myeloid Leukemia

FDA News Release. December 22, 2017.

The U.S. Food and Drug Administration has updated the product label for the cancer drug Tasigna (nilotinib) to include information for providers about how to discontinue the drug in certain patients. Tasigna, first approved by the FDA in 2007, is indicated for the treatment of patients with Philadelphia chromosome-positive (Ph+) chronic myeloid leukaemia (CML). With today’s updated dosing recommendations, patients with early (chronic) phase CML who have been taking Tasigna for three years or more, and whose leukaemia has responded to treatment according to specific criteria as detected by a test that has received FDA marketing authorization, may be eligible to stop taking Tasigna.

“Patients diagnosed with CML generally face a lifetime of treatment to keep their leukaemia from growing or recurring,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “This approval shows that some patients may be able to stop treatment with Tasigna altogether if they are showing a strong response to therapy. While we welcome this progress in patient care, it’s important to note that any discontinuation of treatment still means patients must be regularly monitored for disease recurrence.”

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

Philadelphia chromosome

A certain change in chromosomes (on chromosome 22) found in 95% of patients who have CML. The Philadelphia chromosome results from a mutation that involves the fusion of parts of chromosome 9 and chromosome 22 (the bcr-abl fusion gene)

haematological

Relating to blood or the formation of blood

Also called: hematological

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Chromosome

A chromosome is a structure of DNA that carries the genetic makeup in the nucleus of the cell. Chromosomes contain giant chain molecules of DNA, coiled and folded as aggregates with specific proteins. Chromosomes ensure that during cell division the hereditary information is evenly distributed to the daughter cells. Normal human body cells have 46 chromosomes. Cancer cells can have a different number and/or structure of chromosomes.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Nilotinib

Trade name: Tasigna, development name: AMN107, inhibits BCR-ABL tyrosine kinase. Authorized for marketing in the EU since 2007 for the treatment of CML and Ph+ALL.Other names: |AMN107|Tasigna

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

Ph+

Abbreviation for "Philadelphia-Chromosome-positive", meaning the presence of a certain change in chromosomes (on chromosome 22) found in 95% of patients who have CML. The Philadelphia chromosome results from a mutation that involves the fusion of parts of chromosome 9 and chromosome 22 (the bcr-abl fusion gene).

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.

KEY CONTACT:

BRIEF DESCRIPTION OF ORGANIZATION:

CMl palestine was established for the purpose of serving the CML patients and to raise the knowledge about CML within the community.

The organization was registered recently and in the process of preparing initiatives

KEY OBJECTIVES AND KEY INITIATIVES OF ORGANISATION

The main objetives are:Providing help and advice for CML patientsDistributing knowledge about CMLHelp in regards to drugs and lab tests provisions for patientsCoordination with CML societies localy and internationalyLobbying for the improvement of legislative environment regarding CML treatments

Number of members or patients organisation represents today

7

Chronic Myeloid Leukemia

also called: Chronic Myelogeneous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

Philadelphia chromosome

A certain change in chromosomes (on chromosome 22) found in 95% of patients who have CML. The Philadelphia chromosome results from a mutation that involves the fusion of parts of chromosome 9 and chromosome 22 (the bcr-abl fusion gene)

haematological

Relating to blood or the formation of blood

Also called: hematological

BCR-ABL ratio

The BCR-ABL ratio is a quantitative determination of the CML-typical BCR-ABL gen. The number of BCR-ABL “copies” in relation to a control gen which is present in all cells is a measure of disease activity or "tumour burden" of CML. In lab reports, this value is frequently stated in percent, but sometimes also as a "ratio" or "quotient". In general, the control gen "ABL" is used to calculate the quotient, but other genes may also be used (e.g. BCR, GUSB, G6PD).

Hematologist

A physician who has specialized in blood diseases, including leukemia ("heme" means "blood" in Greek language)

Side effect

Adverse effects of a treatment:, side effects limit the maximum tolerable dose in particular during chemotherapy.

Compliance

Willingness of a patient to reliably adhere to treatment measures and follow medical instructions.

Chromosome

A chromosome is a structure of DNA that carries the genetic makeup in the nucleus of the cell. Chromosomes contain giant chain molecules of DNA, coiled and folded as aggregates with specific proteins. Chromosomes ensure that during cell division the hereditary information is evenly distributed to the daughter cells. Normal human body cells have 46 chromosomes. Cancer cells can have a different number and/or structure of chromosomes.

Dasatinib

Trade name: Sprycel, development name: BMS-354825, inhibits BCR-ABL and SRC tyrosine kinases. Authorized for marketing in the EU since 2006 for the treatment of CML and Ph+ALL.Other names: BMS-354825|BMS354825|Sprycel

Nilotinib

Trade name: Tasigna, development name: AMN107, inhibits BCR-ABL tyrosine kinase. Authorized for marketing in the EU since 2007 for the treatment of CML and Ph+ALL.Other names: |AMN107|Tasigna

Imatinib

Imatinib, trade name Glivec/Gleevec, development name STI-571, a first-generation BCR-ABL tyrosine kinase inhibition. Authorized for marketing since 2002 for the treatment of CML and Ph-positive ALL.Other names: Gleevec|Glivec

BCR-ABL

The abnormal gene that characterizes Chronic Myeloid Leukemia, which is a fusion of the BCR gene of chromosome 9 and the ABL gene of chromosome 22

Chronic

Long-lasting, slowly developping

Acute

Of sudden onset, severe; of short duration.

CLL

Chronic lymphocytic leukemia

TKIs

Tyrosine Kinase Inhibitors, a class of drugs that block an enzyme involved in the mechanism of division of cells

Gene

A unit of information present as DNA; a gene usually contains the blueprint for a protein.

CML

Chronic Myeloid Leukemia, also called Chronic Myelogenous LeukemiaA chronic disease of the blood and bone marrow that results from a transformation of a stem cell.

ASH

American Society of Hematology

MR4

Molecular response, or molecular remission, with a reduction of 4 log (BCR-ABL <0,01%)

Ph+

Abbreviation for "Philadelphia-Chromosome-positive", meaning the presence of a certain change in chromosomes (on chromosome 22) found in 95% of patients who have CML. The Philadelphia chromosome results from a mutation that involves the fusion of parts of chromosome 9 and chromosome 22 (the bcr-abl fusion gene).

CHR

Abbreviation for Complete Hematologic Response. The blood cell count has returned to normal, and tests don’t show any immature white blood cells. Also, the spleen has returned to a normal size if it was enlarged.

FDA

An agency of the United States Department of Health and Human services. The FDA is responsible for protecting and promoting public health through the regulation and supervision of food and drugs.